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Vertex, CRISPR's Gene Editing Drug Gets FDA's Fast Track Tag

Vertex Pharmaceuticals Incorporated
VRTX and partner CRISPR Therapeutics AG CRSP announced that the FDA has assigned Fast Track designation to their gene editing candidate, CTX001, for a second indication. This time the regulatory agency’s prestigious tag is for the treatment of transfusion-dependent beta thalassemia (TDT), a severe genetic disease leading to anemia.

The candidate already has a Fast Track designation for the treatment of sickle cell disease (SCD), a severe hereditary form of anemia, which was granted in January.

The fast track status from the FDA is designed to provide certain benefits to new drugs that treat serious or life-threatening conditions and demonstrate potential to address unmet medical needs. With this designation, the drug may be granted a priority review once it files a new drug application (NDA) or an accelerated approval if the relevant criteria are met.

Shares of CRISPR Therapeutics were up 5.2% on Tuesday in response to the news. In fact, this year so far, CRISPR Therapeutics and Vertex shares have rallied 36.8% and 8.5%, respectively compared with the industry’s increase of 8.4%.

Vertex and CRISPR Therapeutics are co-developing CTX001 in two devastating diseases — TDT and SCD. CTX001 is presently being evaluated in phase I/II studies in TDT in Europe and SCD in the United States. Enrollment in both studies is ongoing.

In December, as part of a strategic research collaboration formed in 2015, Vertex and CRISPR Therapeutics selected CTX001 to move into clinical development as a gene edited treatment for SCD and TDT. The candidate has been developed using CRISPR Therapeutics’ proprietary CRISPR/Cas9 technology. The companies have agreed to co-develop and co-commercialize CTX001 and equally share all R&D costs and profits worldwide. Vertex has rights to license up to six new gene editing treatments (including CTX001), developed using the CRISPR/Cas9 technology, per the joint research deal.

Genomic editing using CRISPR technology to repair defective genetic material that causes diseases is probably one of the most promising and exciting healthcare innovations seen in decades. Though there are several methods to use CRISPR for targeting genetic defects that cause specific diseases, the most promising one is the use of an enzyme called Cas9 to deliver CRISPR to the affected cells.

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